Sarepta Therapeutics Announces Significant Benefits in a
Study for the Treatment of Duchenne

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--Jul 24, 2012 -
CureDuchenne, a nonprofit that raises awareness and funds
research to find a cure for Duchenne muscular dystrophy, is pleased
by today's announcement by
Sarepta Therapeutics (formerly AVI BioPharma) that its
exon-skipping compound, eteplirsen, achieved a significant clinical
benefit in a Phase IIb trial in Duchenne patients. CureDuchenne,
along with Children National Medical Center in Washington, DC and
the Foundation to Eradicate Duchenne, provided funding in 2010 for
this research to progress into human clinical trials.

This is a milestone for the company and patients alike. It
demonstrated for the first time that eteplirsen achieved a highly
significant clinical benefit on the 6-minute walk test, over a
placebo/delayed treatment cohort in a Phase IIb trial in DMD
patients. Eteplirsen was administered once weekly at 50mg/kg over
36 weeks, and patients on treatment demonstrated a 69.4 meter
benefit (227 feet benefit) compared to those who received
placebo/delayed treatment. The drug was well tolerated; there were
no treatment-related adverse events, no serious adverse events and
no discontinuations.

The next milestone is at 48 weeks, and the data is expected in
October. It will include analysis of dystrophin levels from muscle
biopsies in addition to the clinical outcome measures. If it goes
as planned, the result will pave the way for an end of phase II
meeting between the company and the FDA to discuss and agree to the
plan for a phase III registration trial.

“This is a good day for Duchenne, and I'd like to thank
CureDuchenne for their support and funding as well as many other
organizations and institutions that provided support over the last
few years,” said Chris Garabedian, CEO of Sarepta
Therapeutics.

“Our family is so grateful to CureDuchenne for helping to
fund this research which has given us hope for our two boys'
future, for the first time ever,” said Jenn McNary, whose
10-year-old son Max is one of the boys enrolled in the Sarepta
trial. “It is with tears in my eyes that I wish to thank
CureDuchenne, Sarepta, Nationwide Children's Hospital research team
and everyone else who is so dedicated to finding a cure.”

“This is the news the Duchenne community has been waiting
for; a safe and effective disease-modifying treatment is within our
reach and it allows us to focus on the parallel development of next
round of drugs that target the other exons,” said Debra
Miller, founder and CEO of CureDuchenne. “We must remain
cautious since there is still a long way to prove that this drug
will provide significant benefits over time; many drugs fail to
achieve FDA approval at this stage.”

As the most common and lethal form of muscular dystrophy,
Duchenne impacts 1 in 3,500 boys. Boys with Duchenne are usually
diagnosed by the age of 5, in a wheelchair by age 12 and most don't
survive their mid-20s.

About CureDuchenne

CureDuchenne is a national nonprofit organization located in
Newport Beach, Calif., dedicated to finding a cure for Duchenne,
the most common and most lethal form of muscular dystrophy. As the
leading genetic killer of young boys, Duchenne affects more than
300,000 boys worldwide.

CureDuchenne has garnered international attention for its
efforts to raise funds and awareness for Duchenne. With the help of
CureDuchenne's distinguished international panel of Scientific
Advisors, funds raised by CureDuchenne support the most promising
research aimed at treating and curing Duchenne. To date, seven
CureDuchenne research projects have made their way into human
clinical trials – a unique accomplishment as few
health-related nonprofits have been successful in being a catalyst
for human clinical trials.

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